Patients and caregivers: Help us plan the future of UCD research
Apply today to join the NUCDF Partner Network, a new initiative that offers patients, caregivers, and other stakeholders a greater voice in planning the UCD research agenda. Thanks to decades of research, most UCD patients now live longer and have more treatment options. They also face new challenges and questions. As part of the Partner Network, you will work alongside clinicians and researchers, dietitians, front-line care providers, and industry professionals to help us update the research agenda. Together, we will create and share a strategic plan for future UCD research, while building a sustainable infrastructure to carry our work forward.
Partner Network members will:
- Attend a virtual orientation webinar (December 2025)
- Complete five 15-minute online training modules in patient-centered outcomes research (winter 2026)
- Share your thoughts and experiences in a single 90-minute virtual focus group session (summer 2026)
- Complete a brief 10-minute survey (fall 2026)
- Provide brief feedback on a draft roadmap for future research in UCDs (spring 2027)
We will pay members: Up to $290 for full participation over the two-year project.
Why is this project needed?
The urea cycle disorders community is at a critical juncture. Thanks to decades of research, we now have viable treatments. These disorders, once catastrophic, are now complex, chronic conditions for most patients.
However, questions remain about which treatments are right, when, and for which patients. There are still too many missed diagnoses, too little access to expert care, and new challenges arising as patients live longer. Too often, caregivers shoulder overwhelming burdens. Emerging treatments are creating more choices and a greater need for information on which to base decisions.
We want to shift the focus from surviving to thriving, from simply developing treatments to more broadly improving care.
How can I get involved?
All UCD patients and caregivers are invited to apply to join our project now. We are seeking a representative sample of our patient/caregiver community and will select approximately 50 applicants to become Partner Network members. Selected members will be notified later this fall. To be considered, please apply here.
What other stakeholder groups will be joining the Partner Network?
Clinician-researchers, front-line healthcare providers, dietitians, and industry representatives will also be joining the Partner Network. All members will complete the same activities and will be compensated at the same rate.
Can people from outside the United States join the project?
A limited number of international participants may join the project. Please complete our application to express your interest. All participants must have sufficient English-language fluency to participate in a focus group.
What will be the result of the project?
Our project is focused on developing a roadmap for patient-centered research in urea cycle disorders—a strategic plan that outlines the key areas of focus for future research in UCDs. By June 2027, we will share this roadmap and key project findings in scientific and plain language reports, a press release, a Town Hall session, and on a website. All Partner Network members—and the entire UCD community—will be invited to help publicize results.
What will happen after the two-year project ends?
We will transition the Partner Network to a sustainable group that will extend our capacity to engage patients as partners in research into the future. Our long-term goals are to maintain the multi-stakeholder input needed to guide the research agenda, plan and win funding for impactful new research projects, and improve the lives of people affected by UCDs.
How is this project funded?
This project is funded through the Eugene Washington PCORI® Engagement Award Program, an initiative of the Patient-Centered Outcomes Research Institute.
Who is leading this project?
NUCDF staff will lead this project. An Advisory Board that includes representatives of key stakeholder groups is guiding the project.
Who can I contact with questions?
Tresa Warner, NUCDF’s executive director, is the project lead. You can reach her at info@nucdf.org or by calling (626) 578-0833.
